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Déjà Vu: CBER Director’s Intervention Saves Sarepta’s Elevidys Again

 
• By Bridget Silverman

Failure of the EMBARK confirmatory trial convinced US FDA reviewers the Duchenne muscular dystrophy gene therapy did not show clinical benefit, but biologics center director Peter Marks invoked totality of evidence to grant full approval.

Boy in wheelchair
FDA expanded use of Elevidys to non-ambulatory DMD patients under accelerated approval. • Source: Shutterstock

The US Food and Drug Administration’s full approval of Sarepta Therapeutics, Inc.’s Elevidys (delandistrogene moxeparvovec-rokl) pastes another jewel in the company’s “most contentious approvals” crown, with Center for Biologics Evaluation and Research Director Peter Marks again stepping in to over-rule the recommendations of the review team.

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